Eliglustat +/− Imiglucerase for Gaucher Disease

(ELIKIDS Trial)

Not currently recruiting at 74 trial locations

Overseen By Trial Transparency email recommended (Toll free number for US & Canada)

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Sanofi

Must be taking: Enzyme replacement therapy

Must not be taking: Substrate reduction therapy

Disqualifiers: Splenectomy, Transfusion dependent, Cardiac disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of a drug called eliglustat for children with Gaucher disease, a condition where certain fats accumulate due to a missing enzyme. Some participants will use eliglustat alone, while others will combine it with imiglucerase, an enzyme replacement therapy. Children who have been managing Gaucher disease for some time and are already on enzyme replacement therapy might be suitable candidates. The trial aims to determine if eliglustat can improve their health and quality of life. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, you must have been on enzyme replacement therapy for a certain period before joining, and you cannot have used substrate reduction therapy in the last 6 months.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that eliglustat is generally safe for patients with Gaucher disease. One study found that patients who took eliglustat for 4.5 years experienced significant health improvements and had few serious side effects. Another review of several trials with 393 patients found that any side effects were manageable and not life-threatening.

When combined with imiglucerase, studies indicate that this combination is also safe. No new safety issues appeared for children using both treatments together, suggesting that the combination does not increase the risk of side effects.

Overall, current research has shown that both eliglustat alone and with imiglucerase are safe options for treating Gaucher disease. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about these treatments for Gaucher Disease because they offer new approaches to managing the condition. Most treatments for Gaucher Disease involve enzyme replacement therapy (ERT), like imiglucerase, which supplements the deficient enzyme in patients. Eliglustat, however, is a small molecule that inhibits the enzyme glucosylceramide synthase, reducing the production of the harmful substrate glucosylceramide, offering a different mechanism of action. Additionally, the combination of eliglustat with imiglucerase provides a novel treatment strategy that could enhance therapeutic outcomes by targeting the disease through both substrate reduction and enzyme replacement. These innovative approaches have the potential to improve patient outcomes by offering more tailored and possibly more effective treatment options.

What evidence suggests that this trial's treatments could be effective for Gaucher Disease?

Research has shown that eliglustat effectively treats Gaucher disease, a condition where fatty substances accumulate in certain organs. One study found that patients who took eliglustat for 8 years experienced a 69% reduction in spleen size and a 34% reduction in liver size. Their hemoglobin levels and platelet counts also improved, which are important for carrying oxygen and helping blood to clot. In this trial, some participants will receive eliglustat monotherapy, while others will receive a combination of eliglustat and imiglucerase. Research has found that eliglustat alone was just as effective as imiglucerase over 12 months. Patients also reported feeling less tired and having better overall health during treatment. This evidence suggests that eliglustat, with or without imiglucerase, shows promise in managing Gaucher disease. ^{1 2 6 7 8}

Who Is on the Research Team?

Clinical Sciences & Operations

Principal Investigator

Sanofi

Are You a Good Fit for This Trial?

This trial is for kids aged 2-17 with Gaucher Disease types 1 or 3. They must have been on enzyme replacement therapy (ERT) and meet certain health criteria. Girls post-puberty need a negative pregnancy test and agree to contraception or abstinence.

Inclusion Criteria

I am between 2 and 17 years old.

I've been on enzyme replacement therapy for at least 24 months and meet specific treatment goals.

I've been on enzyme replacement therapy for over 36 months and have severe Gaucher's disease symptoms.

Exclusion Criteria

I have been on substrate reduction therapy for Gaucher's disease within the last 6 months.

I have health issues like heart, liver problems, or other serious conditions.

I cannot or do not want to take imiglucerase due to allergies or personal choice.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

8 weeks

Primary Analysis Treatment

Participants receive eliglustat with or without imiglucerase to evaluate safety and pharmacokinetics

52 weeks

Regular visits for monitoring and assessment

Long-term Treatment

Continued treatment with eliglustat or combination therapy based on clinical response

52 weeks

Extension

Participants continue treatment up to Week 364 if clinical benefit is demonstrated

Up to 260 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Eliglustat GZ385660
• Imiglucerase GZ437843

Trial Overview The study tests the safety and effects of Eliglustat alone, or combined with Imiglucerase in children with Gaucher Disease. It looks at how their bodies handle the drugs (pharmacokinetics), changes in their condition, and life quality.

How Is the Trial Designed?

Group I: Cohort 2: Eliglustat plus imiglucerase Experimental Treatment 2 Interventions

Eliglustat plus imiglucerase for three years, at the dose of enzyme replacement therapy received before enrollment. After Week 52, Cohort 2 patients will switch to eliglustat monotherapy for the remainder of the study if the desired clinical response has been achieved.

Group II: Cohort 1: Eliglustat monotherapy Experimental Treatment 1 Intervention

Eliglustat for at least two years. Cohort 1 patients that experience significant clinical decline will receive rescue treatment. Rescue Treatment Step 1: Switch from eliglustat to imiglucerase monotherapy. Rescue Treatment Step 2: Patients who after 6 months of rescue therapy with imiglucerase monotherapy do not show improvement in the parameter(s) that led to the switch from eliglustat to imiglucerase, will then receive combination therapy with eliglustat + imiglucerase.

Who Is Running the Clinical Trial?

Sanofi

Lead Sponsor

Trials

2,246

Recruited

4,085,000+

Paul Hudson

Sanofi

Chief Executive Officer since 2019

Degree in Economics from Manchester Metropolitan University

Christopher Corsico

Sanofi

Chief Medical Officer

MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6587500/

Outcomes after 8 years of eliglustat therapy for Gaucher ... After 8 years of eliglustat, mean spleen and liver volumes decreased by 69% and 34%, respectively. Mean hemoglobin concentration and platelet count increased ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT03485677

NCT03485677 | Safety and Efficacy of Eliglustat With or ... Evaluate the efficacy of eliglustat and quality of life in pediatric patients (≥2 to <18 years old). Detailed Description. The study will include a screening ...

3. sciencedirect.com sciencedirect.com/science/article/abs/pii/S1096719223002974

Patient reported outcomes of patients with Gaucher ... Eliglustat treatment compliance was good and sustained, along with overall health state, fatigue and pain levels, which was consistent with overall patients' ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8457136/

Clinical outcomes after 4.5 years of eliglustat therapy for ... Eliglustat was well‐tolerated and led to clinically significant improvements in previously untreated patients with Gaucher disease type 1 during 4.5 years of ...

5. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/full/10.1002/ajh.27347

Long‐term effectiveness of eliglustat treatment We report real-world outcomes with long-term eliglustat treatment in adults with GD1 in the International Collaborative Gaucher Group Gaucher Registry.

6. sciencedirect.com sciencedirect.com/science/article/pii/S0149291823003120

Effectiveness and Safety of Eliglustat Treatment in Gaucher ... In this study, we evaluated the effectiveness and safety of eliglustat in a cohort of 12 patients with GD followed up in our center.

7. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40166071/

Eliglustat and cardiac comorbidities in Gaucher disease This study represents the first real-world clinical evidence evaluating Eliglustat's cardiac safety in a high-risk GD1 population.

8. ojrd.biomedcentral.com ojrd.biomedcentral.com/articles/10.1186/s13023-019-1085-6

Long-term adverse event profile from four completed trials of ... This pooled analysis of treatment-emergent adverse events combines data from four completed eliglustat clinical trials involving 393 Gaucher disease type 1 ...